Clinical Trial: Search for Serum/Plasma Biomarkers in Pompe's Disease

Study Status: Not yet recruiting
Recruit Status: Not yet recruiting
Study Type: Observational




Official Title: Search for Serum/Plasma Biomarkers in Pompe's Disease

Brief Summary: This study aims to analyze serum and plasma samples from patients with late onset Pompe disease treated and not treated with enzyme replacement therapy (ERT) to identify microRNA that could be specific of the disease. The investigators will correlate the concentration of these microRNA with several muscle function tests and quantitative muscle MRI to know whether they are good biomarkers of progression.